The first real world clinical trial testing is initiated in Europe for the gene editing technology “CRISPR”. The Europe based clinical trial is initiated in late 2018 for blood disorders sickle cell anemia or beta-thalassemia. As many as 12 patients with these conditions were enrolled for the trials with the likely completion date of the project being in 2022. The first molecular disease identified was the Sickle cell disease and there is no cure found for this disease still. CRISPR technology has provided a ray of hope for this condition.
In the US, the FDA has approved the trial under the sponsorship of Vertex Pharmaceuticals and CRISPR Therapeutics.
Geneticist from North Carolina State University, Mr. Rodolphe Barrangou, have some targets like liver diseases and eye diseases. The clinical trials are expected to be a successful, as these diseases are well understood.
Gene editing involves altering the gene by introducing, removing, shifting or swapping cellular DNA. In 2012, the scientists Jennifer Doudna and Emmanuelle Charpentier re-engineered the Cas9 protein with CRISPR to edit DNA segment with comparative effortlessness. The expensive, and cumbersome nature of the earlier gene editing technique is overcome by the CRISPR, which has a capacity to modify numerous DNA segments at once.
The lack of precision of CRISPR technology to target only the gene of interest is one of the major risk factors. The inadvertent editing of the untargeted DNA segments was found to increase the risk of cancer.
More than the challenges of become proficient at definite practices, uncertain queries about the morals and ethics of gene editing endure.
– Rikitha K Murthy,